TY - JOUR
T1 - The OMERACT core domain set for outcome measures for clinical trials in polymyalgia rheumatica
AU - Mackie, Sarah L.
AU - Twohig, Helen
AU - Neill, Lorna M.
AU - Harrison, Eileen
AU - Shea, Beverley
AU - Black, Rachel J.
AU - Kermani, Tanaz A.
AU - Merkel, Peter A.
AU - Mallen, Christian D.
AU - Buttgereit, Frank
AU - Mukhtyar, Chetan
AU - Simon, Lee S.
AU - Hill, Catherine L.
AU - OMERACT PMR Working Group
PY - 2017/10/1
Y1 - 2017/10/1
N2 - OBJECTIVE: To inform development of a core domain set for outcome measures for clinical trials in polymyalgia rheumatica (PMR), we conducted patient consultations, a systematic review, a Delphi study, and 2 qualitative studies. METHODS: Domains identified by 70% or more of physicians and/or patients in the Delphi study were selected. The conceptual framework derived from the 2 qualitative research studies helped inform the meaning of each domain and its relationship to the others. The draft core domain set was refined by further discussion with patients and physicians who had participated in the Delphi study. At the Outcome Measures in Rheumatology (OMERACT) 2016, the domains were discussed and prioritized by 8 breakout groups. Formal voting took place at the end of the workshop and in the final plenary. RESULTS: Ninety-three percent of voters in the final plenary agreed that the inner core of domains considered mandatory for clinical trials of PMR should consist the following: laboratory markers of systemic inflammation, pain, stiffness, and physical function. Patient's global and fatigue were considered important but not mandatory (outer core). The research agenda included psychological impact, weakness, physical activity, participation, sleep, imaging, and health-related quality of life. CONCLUSION: This core domain set was considered sufficiently well-defined that the next step will be to apply the OMERACT Filter 2.0 Instrument Selection Algorithm to select candidate instruments for a subsequent "deeper dive" into the data. This will allow instruments to be mapped onto each of our core domains to derive a core outcome set for PMR.
AB - OBJECTIVE: To inform development of a core domain set for outcome measures for clinical trials in polymyalgia rheumatica (PMR), we conducted patient consultations, a systematic review, a Delphi study, and 2 qualitative studies. METHODS: Domains identified by 70% or more of physicians and/or patients in the Delphi study were selected. The conceptual framework derived from the 2 qualitative research studies helped inform the meaning of each domain and its relationship to the others. The draft core domain set was refined by further discussion with patients and physicians who had participated in the Delphi study. At the Outcome Measures in Rheumatology (OMERACT) 2016, the domains were discussed and prioritized by 8 breakout groups. Formal voting took place at the end of the workshop and in the final plenary. RESULTS: Ninety-three percent of voters in the final plenary agreed that the inner core of domains considered mandatory for clinical trials of PMR should consist the following: laboratory markers of systemic inflammation, pain, stiffness, and physical function. Patient's global and fatigue were considered important but not mandatory (outer core). The research agenda included psychological impact, weakness, physical activity, participation, sleep, imaging, and health-related quality of life. CONCLUSION: This core domain set was considered sufficiently well-defined that the next step will be to apply the OMERACT Filter 2.0 Instrument Selection Algorithm to select candidate instruments for a subsequent "deeper dive" into the data. This will allow instruments to be mapped onto each of our core domains to derive a core outcome set for PMR.
KW - Antirheumatic Agents/therapeutic use
KW - Clinical Trials as Topic
KW - Delphi Technique
KW - Humans
KW - Outcome Assessment (Health Care)
KW - Polymyalgia Rheumatica/drug therapy
KW - Quality of Life
KW - Research Design
U2 - 10.3899/jrheum.161109
DO - 10.3899/jrheum.161109
M3 - Article
C2 - 28765246
VL - 44
SP - 1515
EP - 1521
JO - Journal of Rheumatology
JF - Journal of Rheumatology
SN - 0315-162X
IS - 10
ER -